E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
refractory epilepsy with partial-onset seizures in children aged from 1 month to < 2 years |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10065336 |
E.1.2 | Term | Partial epilepsy |
E.1.2 | System Organ Class | 100000004852 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the safety and tolerability of 1 year of treatment with ESL in the defined patient population and to perform exploratory analyses of efficacy. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Have an informed consent signed by their parent(s) or guardian(s) before undergoing any study-related activities. - Current treatment with 1 to 2 anti-epileptic drugs (except oxcarbazepine; vagus nerve stimulation if present and functioning will not be counted as an anti-epileptic drug). - Have completed the Evaluation Period in Study 211.
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E.4 | Principal exclusion criteria |
- Diagnosis of treatable seizure aetiology such as metabolic, toxic, and infectious disorders. - Primarily generalised seizures. - Known rapidly progressive neurological disorders (e.g. rapidly progressive brain disease, epilepsy secondary to rapidly progressive cerebral lesion). - Seizures of non-epileptic origin. - Diagnosis with Epileptiform Encephalopathies (Early Myoclonic Encephalopathy, Ohtahara syndrome, West syndrome [current], Dravet’s syndrome, or Lennox-Gastaut syndrome). - Uncontrolled cardiac (including atrioventricular block and other clinically significant electrocardiographic abnormalities), renal, hepatic, endocrine, gastrointestinal, metabolic, haematological, or oncology disorders. - Hypersensitivity to the active substance, to other carboxamide derivatives (e.g. carbamazepine, oxcarbazepine), or to any of the excipients, in particular methyl parahydroxybenzoate (E218) or sulphites. - Relevant clinical laboratory abnormalities (e.g. sodium <130 mmol/L, alanine or aspartate transaminases >2 x the upper limit of normal, white blood cell count <3000 cells/mm3) (measured at Visit 1). - Any other condition or circumstance that, in the opinion of the investigator, could compromise the subject’s ability to comply with the study protocol.
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E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy: • Seizures (date, time, type, and duration) recorded by the parent(s) or guardian(s) in study diaries.
Safety: • Treatment-emergent adverse events (TEAEs) defined as all AEs with onset or worsening after first intake of study treatment until 4 weeks after last intake of study treatment. • Clinical laboratory safety tests: - Biochemistry: sodium, potassium, calcium, creatinine, blood urea nitrogen, aspartate transaminase, alanine transaminase, gamma-glutamyl transferase, albumin, total protein, total bilirubin, and glucose. - Haematology: haemoglobin, haematocrit, erythrocyte count, leucocyte count with differential, and platelet count. • Urinalysis: local dipstick test of pH, specific gravity, protein, blood, glucose, ketones, bilirubin, and urobilinogen. If dipstick results indicate a significant abnormality, then microscopy and other appropriate tests (as needed) will be performed by the central laboratory. • Physical examination, vital signs, neurological examination, and electrocardiogram. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Ath the end of the Trial. |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 12 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Romania |
Czechia |
Italy |
Croatia |
Portugal |
Russian Federation |
Serbia |
Ukraine |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |